Kornimate/medical-research-clean huggingface dataset:
a phase 3 study to evaluate safety and biomarkers of resmetirom (mgl-3196) in patients with non-alcoholic fatty liver disease (nafld), maestro-nafld-open-label-extension (maestro-nafld-ole)

Conditions: Non-Alcoholic Fatty Liver Disease

Summary: a 52-week, multi-center, open-label, active treatment extension study to evaluate safety and tolerability of once daily, oral administration of resmetirom (mgl-3196)


Eligibility criteria:
inclusion criteria: * for patients who completed maestro-nafld-1, completed the week 52 visit, and completed the week 56 visit within 90 days of the extension day 1 visit, and are willing to participate in maestro-nafld-ole and provide written informed consent. * for patients who screen failed maestro-nash, must provide written informed consent for maestro-nafld-ole and must have screened and met all eligibility requirements for maestro-nash within 90 days of the extension day 1 visit for maestro-nafld-ole and have a liver biopsy that is ineligible for maestro-nash within 6 months of the extension day 1 visit. eligible liver biopsy for maestro-nafld-ole must have one of the following results: * nas = 3, steatosis 1, ballooning 1, inflammation 1, or nas = 3, ballooning 0, with f1b, f2 or f3 * nas ≥ 4, at least 1 in all nas components, f1a or f1c, pro-c3 ≤ 14 (nash, but ineligible for maestro-nash) * compensated nash cirrhosis based on biopsy at screening of maestro-nash and baseline of maestro-nafld-ole, including child pugh-a (score 5-6), meld \<12, albumin ≥3.2, and bilirubin \<2 * for patients who screen failed from maestro-nash outcomes and de novo patients, must have documented cp-a/b (score \ =15 based on non-cirrhotic parameters. maestro-nash outcomes screen failures must have screened and met all eligibility requirements for maestro-nash outcomes within 90 days of the extension day 1 visit for maestro-nafld-ole . exclusion criteria: * a history of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to extension day 1. * diagnosis of hepatocellular carcinoma or other carcinomas that may prevent participation in the extension study. * chronic liver diseases * has an active autoimmune disease * any other condition which, in the opinion of the investigator, would impede compliance, hinder completion of the study, compromise the well-being of the patient, or interfere with the study outcomes.

Keywords: nafld nash hyperlipidemia resmetirom thyroid hormone receptor beta hepatic fibrosis nash resolution thyroid hormone receptor agonist cardiovascular dyslipidemia fatty liver disease nonalcoholic steatohepatitis
genotype-phenotype correlations in children and adults with ctnnb1 mutation

Conditions: CTNNB1 Gene Mutation

Summary: the aim of this study is to explore and define the correlations between genotype and phenotype in patients with ctnnb1 mutations as well as monitor and survey the natural history of the ctnnb1 syndrome.

ctnnb1 mutation is linked with autism and other neurodevelopmental disorders. so far, there have been 28 studies published describing 71 patients with this syndrome, which leaves much to be yet discovered. the goal of the study is to reach out to the community and include as many families of patients with ctnnb1 syndrome as possible to create a strong and reliable base of information. the investigators are specifically interested in the correlation between different genotypes and phenotypes and the natural course of the disease. the information gathered with this study will help understand ctnnb1 mutations as well as their effect on patient's overall health and wellbeing. the long-term goal is using this information to help create new treatment options, e.g. gene therapy. the participants will be guided through a detailed questionnaire about the patient's history, prenatal and delivery risk factors, current medical issues and standardized questionnaires on a variety of subjects that constitute the patient's health and daily function. the investigators wil ask the participants to provide results of genetic testing and other diagnostic measures, thus providing the medical community as well as the families of the patients a reliable overview of the syndrome.

Eligibility criteria:
inclusion criteria: * patients with a diagnosed mutation in the ctnnb1 gene. * patients whose parents/caregivers were informed about the aims of the study and have signed the informed consent form. exclusion criteria: * patients who do not have a diagnosed mutation in the cttnb1 gene. * patients whose caregivers have not signed the informed consent form.

Keywords: ctnnb1 gene mutation ctnnb1 syndrome beta-catenin autism spectrum disorder developmental delay
study of the pharmacokinetic action of amantadine and ribavirin in chronic hepatitis c. cinam

Conditions: Chronic Hepatitis C

Summary: peg interferon and ribavirin currently represent the standard approved association for treating patients infected with hepatitis c virus (hcv) . the adjunction of amantadine is expected to gain about 10 % of sustained virological response (svr) . unfortunately, about 50 % of the patients remain relapsers or virological non responders. the main predictive factors of svr are hcv genotype and body weight (bw). the impact of the drug pharmacological properties, particularly those of ribavirin requires complementary studies. this drug has a large distribution volume and its concentrations display large inter-individual variability. two studies performed in hcv patients found no correlation between ribavirin dose adjusted on bw and a single ribavirin time point serum concentration at steady state. the aim of this study is to investigate the pharmacokinetic-pharmacodynamic relationships of ribavirin in hepatitis c patient

the study is conducted in naive patients infected with genotype non 2 non 3 administered peginterferon alpha 2-a (40kd) weekly, and ribavirin with dose adjusted on bw (\ 75 kg 1200 mg/day) for the first three months with adjunction of amantadine 200 mg daily for the following 9 months. plasma concentration profiles of ribavirin were studied after the first dose (d0) and at w12. at each period, blood samples were collected pre-dose and 30 minutes, 1, 1.5, 2, 3, 4, 6, 8, and 10 hours post-dosing. ribavirin concentrations were measured using liquid chromatography-tandem mass spectrometry and ribavirin area under the concentration-timcurves (auc0-10h) were derived from plasma concentrations profiles using the linear trapezoidal rule. virological follow-up was performed at w2, w4, w6, w8, w12, w24 and w72. early virological response was defined by undetectable viral load at w12.

Eligibility criteria:
inclusion criteria: * male and female patients \>18 years of age and \<65 years of age * génotype non2 non3 * chronic liver disease consistent with chronic hepatitis c infection on a biopsy (obtained within the past 24 months) as judged by a local pathologist (metavir \>a1and \>f1) * negative urine or blood pregnancy test (for women of childbearing potential) documented within the 24-hour period prior to the first dose of study drug exclusion criteria: * women with ongoing pregnancy or breast feeding * ifn or ribavirin therapy at any previous time * positive test at screening for anti-hav igm ab, hbsag, anti-hbc igm ab, anti-hiv ab * history or other evidence of a medical condition associated with chronic liver disease other than hcv (e.g., hemochromatosis, autoimmune hepatitis, metabolic liver disease, alcoholic liver disease, toxin exposures) * serum creatinine level \>1.5 times the upper limit of normal at screening * history of severe psychiatric disease, especially depression

Keywords: drug monitoring ribavirin-pharmacokinetic pharmacodynamics chronic hepatitis c non 2 non 3 genotype
study of epidermal growth factor on oral mucositis induced by intensive chemotherapy for hematologic malignancies

Conditions: Oral Mucositis

Summary: the purpose of this study is to determine efficacy and toxicity of recombinant human epidermal growth factor (rhegf) as a preventive drug of oral mucositis during intensive chemotherapy with stem cell transplantation in patients with hematologic malignancies.

oral mucositis is one of the most common adverse events during chemotherapy and affects quality of life of patients receiving chemotherapy in relation to the dose of drugs. however, there is only one drug (palifermin) approved by the us fda for the prevention of oral mucositis and the other methods to prevent or treat oral mucositis are just empirical and lack evidences. the results of recent study demonstrated promising efficacy and minimal toxicity of recombinant human epidermal growth factor (rhegf) as a preventive drug of oral mucositis in head and neck cancer patients undergoing radiotherapy (wu hg, et al. cancer 2009;115(16):3699-3708). this clinical trial is a double-blind randomized prospective single-institutional phase ii study to evaluate efficacy and toxicity of recombinant human epidermal growth factor (rhegf) as a preventive drug of oral mucositis during intensive chemotherapy with stem cell transplantation in patients with hematologic malignancies.

Eligibility criteria:
inclusion criteria: * patients with confirmed diagnosis of hematologic malignancies including acute \& chronic leukemia, lymphoma, plasma cell dyscrasia, myelodysplastic syndrome, aplastic anemia, etc. * patients who are planned to receive high-dose chemotherapy with sct * ecog performance status 0-2 * informed consent exclusion criteria: * patients having previous history of hypersensitivity to this drug or similar drugs * patients having oral ulcer or herpes or severe dental disease at the time of inclusion * patients received chemotherapy, radiotherapy, or surgery within 3 weeks * patients who had finished clinical trials which could affect the results of this trial within 4 weeks or are attending one at the time of inclusion * patients having another diseases which have worse prognosis than patients' hematologic malignancy * patients with major psychotic disorder or drug/alcohol abuser * women who are pregnant or breastfeeding * refusal at patients' will * inappropriate patients according to the investigators' opinion

Keywords: oral mucositis intensive chemotherapy stem cell transplantation epidermal growth factor
vaccine hesitancy in black/african americans with rheumatic diseases

Conditions: Rheumatic Diseases; COVID-19 Vaccine; COVID-19; SLE

Summary: to establish the efficacy of a community-based pol (popular opinion leader) intervention with two different trainings designed to increase covid-19 vaccine and booster uptake and reduce hesitancy among social networks of black individuals with rheumatic conditions. the investigators will also determine the structure and composition of the personal and outreach social networks of pols.

through community-academic partnerships in boston and chicago, investigators will recruit and randomly assign pols who identify as black with rheumatic conditions to two training arms in each city. arms 1 and 2 will receive the same core curriculum addressing covid-19 vaccine hesitancy, safety and efficacy. pols will work in their communities to distributed information they learned in the trainings to members of their social network. the investigators will gather information about these social networks, assessing for differences in characteristics such as size, location, and who is in it (percentage of family, percentage black, percentage with rheumatic conditions).

Eligibility criteria:
inclusion criteria: pols: 18-85 years old; all genders; english-speaking; self-identify as black and/or african descent; received ≥1 dose of a covid-19 vaccine after 8.31.2022 (bivalent booster or updated covid-19 vaccine \[2023-2024\] formula) social network members: at least 18 years of age; all genders; english-speaking or haitian creole or spanish-speaking if the pol also speaks haitian creole or spanish; not have received the updated covid-19 vaccine 2023-2024 formula; recent (within 3 months) covid-19 infection and either undecided or no intention of receiving the vaccine. exclusion criteria: both pols and social network members: under 18 years of age pregnant women institutionalized or incarcerated those who are up to date on covid vaccinations

Keywords: social network community based intervention vaccine uptake popular opinion leaders
a study of efficacy and safety of ianalumab versus placebo in addition to eltrombopag in primary immune thrombocytopenia patients who failed steroids

Conditions: Primary Immune Thrombocytopenia

Summary: the purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong time to treatment failure (ttf) in adults with primary itp who failed previous first-line treatment with steroids.

this is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary itp (platelet count \<30 g/l) who failed previous first-line treatment with corticosteroids. after completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with eltrombopag) followed by the eltrombopag tapering period. afterwards, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how the participants responded to the study treatment.

Eligibility criteria:
key inclusion criteria 1. male or female patients aged 18 years and older on the day of signing the informed consent. 2. a signed informed consent must be obtained prior to participation in the study. 3. a diagnosis of primary itp, with insufficient response to, or relapse after a first-line corticosteroid therapy ± ivig. 4. patient with platelet count \<30g/l (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag key exclusion criteria 1. itp patients who received second-line itp treatments (other than steroid therapy± ivig) including splenectomy. however, patients exposed to thrombopoietin receptor agonists (tpo-ras) for a limited time (max one week) before screening are eligible. 2. patients with key lab abnormalities and patients with evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible). 3. patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters 4. patients with current or history of life-threatening bleeding 5. patient that are human immunodeficiency virus (hiv), hepatitis c virus (hcv), hepatitis b surface antigen (hbsag)/ hepatitis b core antibody (hbcab)-positive. hbcab-positive patients can be enrolled if hbsag negative, hbv dna negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given 6. patients with known active or uncontrolled infection requiring systemic treatment during screening period 7. patients with hepatic impairment 8. patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily) 9. nursing (breast feeding) or pregnant women other protocol-defined inclusion/exclusion criteria may apply.

Keywords: primary immune thrombocytopenia (itp), ianalumab vay736 b-cell depletion b-cell activating factor receptor (baff-r) blockade eltrombopag
safety of estrogens in lupus: hormone replacement therapy

Conditions: Systemic Lupus Erythematosus

Summary: safety of estrogens in lupus erythematosus - national assessment (selena) is a study to test whether postmenopausal women with systemic lupus erythematosus (sle, or lupus) can safely use the hormone estrogen. in this part of the study, we will look at the effects of estrogen replacement therapy on the activity and severity of disease in women with sle.

this study tests the effect of exogenous female hormones on disease activity and severity in women with systemic lupus erythematosus (sle). physicians generally do not prescribe hormone replacement therapy (hrt) to women with sle because of the widely held view that such treatment can activate sle. this practice is based on the greater incidence of sle in women than in men, biologic abnormalities of estrogen metabolism, murine models of lupus, several anecdotes of patients having disease flares while receiving exogenous hormones, and a single retrospective study in patients with preexisting renal disease. by contrast, recent retrospective studies suggest that the rate of flare is not significantly increased in patients taking hrt. we will examine, in a multicenter, randomized, double-blind, placebo-controlled trial, the effect of hormonal replacement with conjugated estrogens on disease activity in postmenopausal women with sle. we will recruit patients from clinics and private practices that include over 4,000 women with sle, most belonging to minority groups. we will give patients hormones for 1 year. note: this trial has been terminated as of august 2002 upon recommendation of the data safety monitoring board (dsmb), based on the findings of the whi trial. study subjects have discontinued study drug but will continue followup visits to study doctors through may 2003

Eligibility criteria:
inclusion criteria: * female * unequivocal diagnosis of sle * inactive disease or stable on 0.5 mg/kg/day or less of prednisone * chemical evidence of menopause or have stopped periods for at least 6 months exclusion criteria: * blood pressure \>145/95 on three occasions * deep vein, arterial thrombosis or pulmonary embolus * gpl \>40; mpl \>40; apl \>50; drvvt \>37 sec * apl antibody syndrome ever * gynecologic or breast cancer * hepatic dysfunction or liver tumors * diabetes mellitus (not due to steroids) with vascular disease * congenital hyperlipidemia * complicated migraine * severe disease activity (sledai \>12) * increase in sledai \>2 points in 3 months * unexplained vaginal bleeding * use of estrogen (hrt or ocp) for \>1 month at any time after sle diagnosis * fsh \<40 * premenopausal myocardial infarction

Keywords: sle selena estrogen replacement therapy (ert) postmenopause osteoporosis estrogens steroids progestin placebo
safety and effectiveness of giving adefovir dipivoxil plus abacavir plus efavirenz plus amprenavir to hiv-infected patients who have failed to respond to previous protease inhibitor treatment

Conditions: HIV Infections

Summary: the purpose of this study is to see if it is safe and effective to give adefovir dipivoxil plus abacavir (abc) plus efavirenz (efv) plus amprenavir (apv) to hiv-infected patients who have failed to respond to previous treatment with protease inhibitors (pis).

patients receive a treatment regimen of adefovir dipivoxil, abacavir, efavirenz, and amprenavir for 24 weeks. during the study, patients are evaluated for changes from baseline in plasma hiv-1 rna levels and lymphocyte subsets and for development of adverse events and toxicities. patients who experience virologic failure are discontinued from the study. after week 24, patients with documented virologic response are eligible to continue receiving the study treatment and to attend scheduled follow-up visits.

Eligibility criteria:
inclusion criteria you may be eligible for this study if you: * are hiv-positive. * had an hiv level of at least 2,000 copies/ml after 6 months of treatment with at least 1 pi other than amprenavir (meaning you failed pi treatment). * are at least 13 years old (need consent of parent or guardian if under 18). * are able to complete the study. * agree to use effective barrier methods of birth control, such as condoms, during the study. exclusion criteria you will not be eligible for this study if you: * have certain serious medical conditions, including aids-related cancers (except kaposi's sarcoma) that require treatment during the study. * have ever taken or are allergic to adefovir dipivoxil, abc, apv, efv. * are participating in another anti-hiv drug trial during this study. * have taken certain medications within 30 days prior to study entry, including medications that affect your immune system. * have been diagnosed with hepatitis within the past 30 days. * abuse alcohol or drugs. * are pregnant or breast-feeding. * have ever taken nnrtis. * have ever taken ddi or d4t. * have received chemotherapy or radiation therapy within 30 days prior to study entry.

Keywords: drug therapy, combination hiv protease inhibitors reverse transcriptase inhibitors anti-hiv agents
evaluation of an intervention to promote physical activity in outpatients with mental disorders

Conditions: Mental Disorders

Summary: the purpose of this study is to determine if a manualised intervention to promote physical activity (movo-lisa) is effective to help psychiatric outpatients to increase their level of everyday physical activity.

the intervention program movo-lisa is a psychological intervention that should help participants to increase their level of physical activity for a healthy lifestyle.it contains three sessions (2 group,1 one on one) in the course of two weeks. the intervention focuses on planning and barrier management. participants develop activity ideas, goals and plans that enable them to integrate physical activity in their everyday routine and how to shield their motivation against barriers and difficulties.the intervention has proven to be effective with patients with pain disorders in rehabilitation. this study examines if the intervention if effective for psychiatric outpatients as well. the intervention is compared to an active control group that receives a similar program that is focusing on a healthy diet. participants are recruited in the outpatient departements of three hospitals in berlin and brandenburg (charité universitätsmedizin berlin - departement of psychiatry and psychotherapy; st. hewdig hospital berlin - departement of psychiatry; oberhavel hospital brandenburg, departement of psychiatry) and at local psychiatrists in berlin. individuals are recruited by posters and flyers in the waiting areas areas or by information of their doctor. patients can contact the researcher and are, after checked for eligibility, invited to an information event, where they get detailed information on the program and the study. after the information event, participants give written informed consent. the intervention takes place in the outpatient departements of the three hospitals. clinical information about diagnoses, medication and functioning is obtained by their doctors by mail after the patients released them from confidentiality. the study is an explorative approach to test if the program is suited for psychiatric patients. a sample size of 50 participants that complete the program is planned. all data is collected pseudonymised to protect the identity of the patients.

Eligibility criteria:
inclusion criteria: * over 18 years of age * patients in one of the psychiatric outpatients or at an local psychiatrist in berlin * mental illness (icd-10: f1-f4) * no contraindications for physical activity * able to understand german language exclusion criteria: * contraindications to physical activity * acute suicidality

Keywords: physical activity mental health physical exercise mental disorder promotion